# US 2014/0228301 A1 · Recombinant human alpha-1-antitrypsin for the treatment of inflammatory disorders

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- Number: US 2014/0228301 A1
- Document ID: US20140228301A1
- Status: Published application
- Kind: A1
- Application number: 14/342,170
- Filed: 2012-12-19
- Published: 2014-08-14
- Earliest claimed: 2011-12-19
- Assignees: rEVO Biologics, Inc.
- Applicants: rEVO Biologics, Inc.
- Inventors: Harry M. Meade; Paul R. Bourdon
- Web page: https://patentgrep.com/patent/US20140228301A1

## CPC

- A61K 38/57: from animals; from humans {(A61K38/553, A61K38/556 take precedence)}
- A01K 67/0275: Genetically modified vertebrates, e.g. transgenic
- A61P 11/00: Drugs for disorders of the respiratory system
- A61P 29/00: Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
- A61P 43/00: Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
- C07K 14/8125: {Alpha-1-antitrypsin}
- A01K 2217/052: inducing gain of function
- A01K 2227/105: Murine
- A01K 2267/01: Animal expressing industrially exogenous proteins
- A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D)
- A01K: ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- C07K: PEPTIDES (peptides in foodstuffs A23; obtaining protein compositions for foodstuffs, working-up proteins for foodstuffs A23J; preparations for medicinal purposes A61K; peptides containing beta-lactam rings C07D; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, C07D; ergot alkaloids of the cyclic peptide type C07D519/02; macromolecular compounds having statistically distributed amino acid units in their molecules, i.e. when the preparation does not provide for a specific; but for a random sequence of the amino acid units, homopolyamides and block copolyamides derived from amino acids C08G69/00; macromolecular products derived from proteins C08H1/00; preparation of glue or gelatine C09H; single cell proteins, enzymes C12N; genetic engineering processes for obtaining peptides C12N15/00; compositions for measuring or testing processes involving enzymes C12Q; investigation or analysis of biological material G01N33/00)

## Abstract

In one aspect, the disclosure relates to compositions comprising alpha-1-antitrypsin (AAT) and the production thereof. In some embodiments, the AAT is recombinantly produced. The disclosure also relates to methods of administering compositions comprising alpha-1-antitrypsin (AAT).

## Claims

### Claim 1

A composition comprising alpha-1-antitrypsin (AAT), wherein the AAT is recombinantly produced.

### Claim 2 (depends on 1)

The composition of claim 1, wherein the AAT is produced in mammary epithelial cells of a non-human mammal.

### Claim 3 (depends on 1)

The composition of claim 1, wherein the AAT is produced in a transgenic non-human mammal.

### Claim 4 (depends on 2, 3)

The composition of claim 2 or claim 3, wherein the non-human mammal is a goat, sheep, bison, camel, cow, pig, rabbit, buffalo, horse, rat, mouse or llama.

### Claim 5 (depends on 4)

The composition of claim 4, wherein the non-human mammal is a goat.

### Claim 6 (depends on 1, 2, 3, 4, 5)

The composition of any one of claims 1-5, wherein the recombinantly produced AAT has enhanced deoxyhexose glycosylation compared to plasma-derived AAT.

### Claim 7 (depends on 1, 2, 3, 4, 5, 6)

The composition of any one of claims 1-6, wherein the recombinantly produced AAT has been modified to increase the sialylation on the AAT-glyco-motifs.

### Claim 8

A composition comprising AAT wherein the AAT has a high level of deoxyhexose glycosylation.

### Claim 9

A composition comprising AAT wherein the AAT has a high level of sialylation on the AAT-glyco-motifs.

### Claim 10

A composition comprising AAT wherein the AAT has a high level of deoxyhexose glycosylation and a high level of sialylation on the AAT-glyco-motifs.

### Claim 11 (depends on 1, 2, 3, 4, 5, 6, 7, 8, 9, 10)

A composition comprising the AAT of any one of claims 1-10, further comprising milk.

### Claim 12 (depends on 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11)

A composition comprising the AAT of any one of claims 1-11, further comprising a pharmaceutically acceptable carrier.

### Claim 13 (depends on 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12)

Mammary gland epithelial cells that produce the AAT of the compositions of any one of claims 1-12.

### Claim 14 (depends on 13)

A transgenic non-human mammal comprising the mammary gland epithelial cells of claim 13.

### Claim 15 (depends on 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12)

A method comprising administering the composition of any one of claims 1-12 to a subject in need thereof.

### Claim 16 (depends on 15)

The method of claim 15, wherein the subject has alpha-1-antitrypsin deficiency.

### Claim 17 (depends on 15)

The method of claim 15, wherein the subject has an inflammatory disorder.

### Claim 18 (depends on 17)

The method of claim 17, wherein the inflammatory disorder is emphysema.

### Claim 19 (depends on 15, 16, 17, 18)

The method of any one of claims 15-18, wherein the composition is administered at a dose of from 30 mg/kg to about 60 mg/kg AAT.

### Claim 20 (depends on 15, 16, 17, 18, 19)

The method of any one of claims 15-19, wherein the composition is administered intravenously.

### Claim 21 (depends on 15, 16, 17, 18, 19)

The method of any one of claims 15-19, wherein the composition is administered by inhalation.

### Claim 22 (depends on 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12)

A method of reducing elastase activity in the lung, the method comprising administering the composition of any one of claims 1-12 to a subject in an amount sufficient to reduce elastase activity in the lung.